Roche has shared positive five-year results for its spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam) in children.
Affecting approximately one in every 10,000 babies, SMA is a severe and progressive neuromuscular disease characterised by insufficient levels of the SMN protein.
Depending on the form of SMA, patients’ physical strength and their ability to walk, eat or breathe can be considerably diminished or lost.
Roche’s Evrysdi, which can be administered at home in liquid form by mouth or by feeding tube, is designed to treat the disease by increasing and sustaining the production of SMN protein in the central nervous system and peripheral tissues.
The open-label extension of the FIREFISH study has been evaluating the safety and efficacy of the SMN2 splicing modifier in children with type 1 SMA who, without treatment, are not expected to live past two years and are never able to sit without support.
After five years of treatment with Evrysdi, 91% of patients were alive, 81% were alive without permanent ventilation and 59% were able to sit without support for at least 30 seconds.
The long-term data presented at the Cure SMA Research and Clinical Care Meeting also showed that seven children were able to stand, three with support, four unaided and six could walk with support by the end of year five.
Motor function abilities were maintained or continued to be achieved in those treated with Evrysdi, and 96% of those assessed at year five were able to swallow and 80% were able to feed without a feeding tube.
Levi Garraway, chief medical officer and head of global product development, outlined that the latest readout is the last for FIREFISH, adding that the study has “provided a wealth of insights and data, helping to firmly establish Evrysdi as an important treatment option, improving the lives of children across the globe living with SMA”.
Roche leads the clinical development of Evrysdi, which is already approved in more than 100 countries, as part of a collaboration with the SMA Foundation and PTC Therapeutics. Submissions for Evrysdi are also currently under review in a further 13 countries.